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ACTIVE NOT RECRUITING

NCT03876769

PHASE2

Study of Efficacy and Safety of Tisagenlecleucel in HR B-ALL EOC MRD Positive Patients

Sponsor: Novartis Pharmaceuticals

View on ClinicalTrials.gov

Summary

This is a single arm, open-label, multi-center, phase II study to determine the efficacy and safety of tisagenlecleucel in de novo HR pediatric and young adult B-ALL patients who received first-line treatment and are EOC MRD positive. The study will have the following sequential phases: screening, pre-treatment, treatment \& follow-up, and survival. After tisagenlecleucel infusion, patient will have assessments performed more frequently in the first month and then at Day 29, then every 3 months for the first year, every 6 months for the second year, then yearly until the end of the study. Efficacy and safety will be assessed at study visits and as clinically indicated throughout the study. The study is expected to end in approximately 8 years after first patient first treatment (FPFT). A post-study long term follow-up safety will continue under a separate protocol per health authority guidelines.

Official title: A Phase II Trial of Tisagenlecleucel in First-line High-risk (HR) Pediatric and Young Adult Patients With B-cell Acute Lymphoblastic Leukemia (B-ALL) Who Are Minimal Residual Disease (MRD) Positive at the End of Consolidation (EOC) Therapy

Key Details

Gender

All

Age Range

1 Year - 25 Years

Study Type

INTERVENTIONAL

Enrollment

121

Start Date

2019-06-24

Completion Date

2027-10-19

Last Updated

2026-03-13

Healthy Volunteers

Conditions

B-Cell Acute Lymphoblastic Leukemia

Interventions

BIOLOGICAL

CTL019

Based on the subject's weight, one of two possible dose ranges will be prepared for the subject: Subjects ≤ 50 kg: 0.2 to 5.0 x 10(6) CAR-positive viable T cells per kg body weight OR Subjects \> 50 kg: 0.1 to 2.5 x 10(8) CAR-positive viable T cells

Inclusion Criteria: 1. CD19 expressing B-cell Acute Lymphoblastic Leukemia 2. De novo NCI HR B-ALL who received first-line treatment and are MRD ≥ 0.01% at EOC. EOC bone marrow MRD will be collected prior to screening and will be assessed by multi-parameter flow cytometry using central laboratory analysis. 3. Age 1 to 25 years at the time of screening 4. Lansky (age \< 16 years) or Karnofsky (age ≥ 16 years) performance status ≥ 60% 5. Adequate organ function during the screening period: A. Renal function based on age/gender B. ALT ≤ 5 times ULN for age C. AST ≤ 5 times ULN for age D. Total bilirubin \< 2 mg/dL (for Gilbert's Syndrome subjects total bilirubin \< 4 mg/dL) E. Adequate pulmonary function defined as: * no or mild dyspnea (≤ Grade 1) * oxygen saturation of \> 90% on room air F. Adequate cardiac function defined as LVSF ≥ 28% confirmed by echocardiogram or LVEF ≥ 45% confirmed by echocardiogram or MUGA within 6 weeks of screening 6. Prior induction and consolidation chemotherapy allowed: 1st line subjects: ≤ 3 blocks of standard chemotherapy for first-line B-ALL, defined as 4-drug induction, Berlin-Frankfurt-Münster (BFM) consolidation or Phase 1b, and interim maintenance with high-dose methotrexate. Exclusion Criteria: 1. M3 marrow at the completion of 1st line induction therapy 2. M2 or M3 marrow or persistent extramedullary disease at the completion of first-line consolidation therapy or evidence of disease progression in the peripheral blood or new extramedullary disease prior to enrollment. Patients with previous CNS disease are eligible if there is no active CNS involvement of leukemia at the time of screening. 3. Philadelphia chromosome positive ALL 4. Hypodiploid: less than 44 chromosomes and/or DNA index \< 0.81, or other clear evidence of a hypodiploid clone 5. Prior tyrosine kinase inhibitor therapy 6. Subjects with concomitant genetic syndromes associated with bone marrow failure states: such as subjects with Fanconi anemia, Kostmann syndrome, Shwachman syndrome or any other known bone marrow failure syndrome. Subjects with Down syndrome will not be excluded. 7. Subjects with Burkitt's lymphoma/leukemia (i.e. subjects with mature B-ALL, leukemia with B-cell \[sIg positive and kappa or lambda restricted positivity\] ALL, with FAB L3 morphology and /or a MYC translocation) 8. Has had treatment with any prior anti-CD19 therapy 9. Treatment with any prior gene or engineered T cell therapy Other protocol-defined inclusion/exclusion may apply.

Locations (45)

Children s Hospital of Alabama

Birmingham, Alabama, United States

Phoenix Childrens Hospital

Phoenix, Arizona, United States

City of Hope National Medical

Duarte, California, United States

Childrens Hospital Los Angeles

Los Angeles, California, United States

Mattel Childrens Hospital UCLA

Los Angeles, California, United States

Childrens Hospital of Orange County

Orange, California, United States

Rady Children s Hospital

San Diego, California, United States

UCSF Medical Center

San Francisco, California, United States

Stanford University Medical Center

Stanford, California, United States

Childrens Hospital Colorado

Aurora, Colorado, United States

Childrens National Hospital

Washington D.C., District of Columbia, United States

Johns Hopkins All Childrens

St. Petersburg, Florida, United States

Childrens Healthcare of Atlanta

Atlanta, Georgia, United States

James Whitcomb Riley Hospital For Children

Indianapolis, Indiana, United States

Johns Hopkins Oncology Center

Baltimore, Maryland, United States

Dana Farber Cancer Institute

Boston, Massachusetts, United States

Children s Mercy Hospital

Kansas City, Missouri, United States

Hackensack Uni Medical Center

Hackensack, New Jersey, United States

Roswell Park Cancer Institute

Buffalo, New York, United States

Memorial Sloan Kettering Cancer Ctr

New York, New York, United States

Columbia University Medical Center

New York, New York, United States

Duke University Medical Center

Durham, North Carolina, United States

Cinn Children Hosp Medical Center

Cincinnati, Ohio, United States

Oregon Health and Science University

Portland, Oregon, United States

The Childrens Hosp of Philadelphia

Philadelphia, Pennsylvania, United States

Univ of Texas Southwest Med Center

Dallas, Texas, United States

Texas Childrens Cancer and Hematology Center

Houston, Texas, United States

Methodist Childrens Hospital

San Antonio, Texas, United States

University of Utah Clinical Trials Office

Salt Lake City, Utah, United States

University of Wisconsin Hospital and Clinics Pharmacy/Drug Shipping Address

Madison, Wisconsin, United States

Childrens Hospital of Wisconsin

Milwaukee, Wisconsin, United States

Novartis Investigative Site

Ghent, Belgium

Novartis Investigative Site

Calgary, Alberta, Canada

Novartis Investigative Site

Toronto, Ontario, Canada

Novartis Investigative Site

Montreal, Quebec, Canada

Novartis Investigative Site

Copenhagen, Denmark

Novartis Investigative Site

Paris, France

Novartis Investigative Site

Roma, RM, Italy

Prinses Maxima Centrum voor Kinderoncologie

Utrecht, CS, Netherlands

Novartis Investigative Site

Utrecht, Netherlands

Novartis Investigative Site

Oslo, Norway

Novartis Investigative Site

Esplugues, Barcelona, Spain

Novartis Investigative Site

Gothenburg, Sweden

Novartis Investigative Site

London, United Kingdom

Novartis Investigative Site

London, United Kingdom

Clinical Research Directory

Study of Efficacy and Safety of Tisagenlecleucel in HR B-ALL EOC MRD Positive Patients

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (45)